Indo-US Research Conference on Rare Diseases and Orphan Drugs

描述（由申请人提供）： 据估计，有2500万至3000万美国人患有罕见疾病，根据《孤儿药法》的定义，罕见疾病的患病率为20万或更低。数亿人，主要是在发展中国家，患有相对常见的医疗问题，这些问题几乎没有有效的治疗方法。这些被称为被忽视的疾病，因为大多数被政府机构和生物医学行业忽视。一些被忽视的疾病，如疟疾和肺结核，也是美国日益严重的公共卫生问题。罕见疾病和被忽视的疾病一起造成巨大痛苦，并在医疗费用和生产力损失方面给社会造成非常高的代价。生物医学研究为科学家提供了对罕见疾病和可用于开发新疗法的潜在靶点的更深入了解，这些新疗法被称为孤儿药和生物制剂。然而，由于药物开发失败率高以及与罕见疾病相关的市场小，制药业一直不愿利用这些机会。为了解决这一难题，政府和非政府组织正在努力吸引学术界科学家参与孤儿药研究。需要科学家之间的全球合作努力，以充分利用开发罕见疾病新疗法的潜力。拟议的会议旨在汇集来自印度和美国的广泛的翻译和临床研究人员，他们对罕见病和孤儿药感兴趣，分享他们的研究并促进合作。 公共卫生相关性：为期三天的会议名为“印度-美国罕见病和孤儿药会议”，与会者将分享他们的研究和专业知识，了解NIH和FDA的计划，并更好地了解与孤儿药开发相关的商业化和监管问题。会议成果包括：1）更好地了解罕见和被忽视疾病的研究机会，2）建立新的国际合作，3）在印度等发展中国家启动孤儿药的发现和开发。该会议将成为未来会议的典范，旨在加速全球罕见和被忽视的医学问题的新疗法的开发。罕见和被忽视的疾病是一个重大的全球公共问题。美国有数百万人，全世界有数亿人患有几乎没有安全有效的治疗方法的疾病。印度-美国罕见病和孤儿药研究会议将汇集来自印度和美国的顶尖科学家，分享研究成果，建立合作，并提高对孤儿药和生物制剂开发需求的认识，特别是在发展中国家。

DESCRIPTION (provided by applicant): An estimated 25-30 million Americans suffer from rare disorders, which are defined in the Orphan Drug Act as having prevalence of 200,000 or less. Hundreds of millions, primarily in developing countries, have relatively common medical problems for which there are few, if any, effective therapies. These have been dubbed neglected diseases because most have been ignored by governmental agencies and the biomedical industry. Some neglected diseases such as malaria and tuberculosis are also a growing public health concern in the United States. Together rare and neglected diseases cause enormous suffering and exact a very high cost to society in terms of medical expenses and lost productivity. Biomedical research is providing scientists with a greater understanding of rare diseases and the potential targets that can be exploited for development of new therapies, known as of orphan drugs and biologics. Industry, however, has been reluctant to capitalize on these opportunities because of the high drug development failure rate and the small markets associated with rare diseases. In an effort to address this dilemma, governmental and non-governmental organizations are seeking to attract academic scientists to orphan drug research. A global, collaborative effort among scientists is needed to fully leverage the potential to develop new therapies for rare diseases. The proposed conference is designed to bring together a wide range of translational and clinical researchers from India and the US with interests in rare diseases and orphan drugs to share their research and foster collaborations. PUBLIC HEALTH RELEVANCE: A three day conference entitled "Indo-US Conference on Rare Diseases and Orphan Drugs" will allow attendees to share their research and expertise, learn about NIH and FDA programs, and gain a better understanding of the commercialization and regulatory issues related to the orphan drug development. Conference outcomes include: 1) a better understanding of research opportunities in rare and neglected diseases, 2) establishment of new international collaborations, and 3) initiation of orphan drug discovery and development in developing countries such as India. The conference will serve as a model for future conferences intended to accelerate the worldwide development of new therapies for rare and neglected medical problems. Rare and neglected diseases are a major, worldwide public issue. Millions in the US and hundreds of millions worldwide suffer from disorders for which there are few, if any, safe and effective therapies. The Indo-US Research Conference on Rare Diseases and Orphan Drugs will bring together leading scientists from India and the US to share research, build collaborations, and increase awareness of the need for the development of orphan drugs and biologics particularly in developing countries.